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A Comprehensive Procedure to Evaluate the In Vivo Performance of Cancer Nanomedicines
07:59

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A review of study designs and outcomes of phase I clinical studies of nanoparticle agents compared with

Whitney P Caron1, Katherine P Morgan, Beth A Zamboni

  • 1Division of Pharmacotherapy and Experimental Therapeutics, University of North Carolina (UNC) Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, , Chapel Hill, NC 27599, USA.

Clinical Cancer Research : an Official Journal of the American Association for Cancer Research
|April 27, 2013
PubMed
Abstract

Insights

Nanoparticle anticancer agents allow for greater dose escalation in clinical trials compared to small-molecule drugs. This highlights the need for better preclinical animal models to assess nanoparticle toxicity.

Area of Science:

  • Oncology
  • Nanomedicine
  • Pharmacology

Background:

  • Nanoparticles offer improved drug delivery and circulation times over small molecules.
  • Current phase I study dosing for anticancer agents relies on preclinical toxicity, but optimal models for nanoparticles are unknown.

Purpose of the Study:

  • To compare the design, progression, and outcomes of phase I clinical trials for nanoparticle-based versus small-molecule anticancer agents.
  • To evaluate the dose escalation potential and safety profiles of nanoparticles in early-phase clinical studies.

Main Methods:

  • Preclinical assessment of maximum tolerated dose (MTD) in rats and dogs for nanoparticles and small molecules.
  • Analysis of phase I clinical trial data, including starting dose, dose escalation, patient enrollment, and MTD-to-starting dose ratios.

Main Results:

  • Nanoparticle studies showed a significantly higher mean MTD-to-starting dose ratio (13.9) compared to small molecules (2.1).
  • Phase I trials for nanoparticles involved significantly more dose levels (7.3) than those for small molecules (4.1).

Conclusions:

  • Nanoparticle anticancer drugs exhibit a greater degree of dose escalation from the starting dose to the MTD than small-molecule drugs.
  • These findings underscore the critical need to identify the most suitable preclinical animal models for evaluating nanoparticle toxicity.

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